NIH Begins Gene Therapy Trial for Parkinson’s Disease

All eyes were on Perry Cohen when he froze at the microphone. His voice failed him. He couldn’t read his notes. Eventually, the once-powerful Parkinson’s disease speaker had to be helped off the stage halfway through his speech.

That was in February 2012, but the memory of that day is emblazoned in his mind. “It was the adrenaline and the pressure of speaking – it drained all the dopamine out,” Cohen says, referring to the brain chemical that is found lacking in the neurodegenerative disorder. “That’s why my symptoms got worse.” When Cohen learned he had Parkinson’s disease 17 years ago his symptoms were subtle. In the past couple years, however, the deterioration of his nervous system has become increasingly obvious, ultimately threatening to silence one of the most prominent voices in the Parkinson’s patient community.

Cohen is now first in line to try a novel treatment he hopes will halt or even reverse the symptoms of his Parkinson’s disease. Two months ago he became the inaugural patient to undergo a gene therapy treatment led by the National Institutes of Health.

The trial attempts to devise an intervention for Parkinson’s disease at the root of the problem: protecting dopamine in the brain. Researchers in this trial are attempting to surgically deliver a gene into the body that will make a natural protein to protect dopaminergic neurons, the brain cells attacked by the disease. To date no Parkinson’s treatment is geared toward reversing the progression of Parkinson’s disease.

For patients in the Parkinson’s community Cohen has become a squeaky wheel that cannot be ignored, helping researchers understand more about the patient experience and how to utilize their input in procedures and patient care.A scientist with a PhD from Massachusetts Institute of Technology in organization studies, Cohen says his background helped fuel his ability to bring people together to take action to advocate for people with Parkinson’s disease. His persistency also helped keep him on researchers’ radars when they were hunting for eligible patients for the trial…